To mark Rare Disease Day 2024, this week we are exploring four critical elements to sustaining rare disease innovation in Europe. Today focuses on requirement #3: Supportive P&R. Whether a medicine exists matters little for patients who cannot receive it because it is not reimbursed. Reimbursement is also key for manufacturers who rely on the sale of medicines to recover their considerable investment in development and to support their future pipeline. Continued innovation and access in rare disease in Europe relies on country-level pricing and reimbursement (P&R) approaches that can recognise and reward promising orphan developments. But this is by no means simple since many extremely novel, transformative treatments fall foul of traditional P&R systems designed for chronic therapies in common conditions. One example is cell and gene therapies. These unique treatments present immense value owing to their one-time, potentially curative profile. They also come with a significant price tag, complex delivery, and an inevitable degree of uncertainty around long-term benefit. In our recent paper for the Alliance for Regenerative Medicines (ARM), Dolon's Managing Director, Adam Hutchings, explores these challenges and their implications for P&R, patient access, and the sustainability of ATMP innovation in Europe. In particular, we focus on innovative contracting as an opportunity to mitigate these challenges as well as recent learnings from manufacturers’ experience in negotiating these. P&R frameworks need to evolve with science if they are to fully reflect the value of emerging therapies to patients and health systems alike. Failure to do so will come at the expense of patients and health systems today, and Europe’s standing as a global centre for innovation in the longer term. Read on to learn more: https://bit.ly/42UC0Uf #RareDiseaseDay2024 #RareDiseaseDay #RDD2024 #LightUpForRare #ShareYourColours #RareDiseases #RareDiseaseAwareness #RareDiseaseInnovation #Dolon
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May is #LupusAwarenessMonth! At Alumis, we're committed to improving the lives of people living with lupus by shedding light on this complex autoimmune disease and advancing clinical research. Recognizing the disparities in #lupus care, especially in under-resourced regions, we’re proud to be a member of the Lupus Research Alliance 's Lupus Accelerating Breakthroughs Consortium (Lupus ABC), to pioneer better endpoints and advance more impactful clinical trials. Learn more how you can get involved in supporting the lupus community: https://lnkd.in/esmUVvvw #LupusAwareness #LupusResearch
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📣 NEW! LightCure Consortium receives Horizon Europe Grant. 💡 Congenital Hyperinsulinism International (CHI) is excited to announce we are part of a new 11-member research consortium called LightCure. The LightCure Consortium is developing a targeted photodynamic therapy, that one day could be a promising treatment for congenital hyperinsulinism (HI). 💙 This project is made possible by a 72-month-long grant of just over €8.2M from the European Union research arm Horizon Europe and encompasses CHI’s work to strengthen patient engagement and raise worldwide awareness of HI and its myriad challenges. 🔗 You can find more information about this exciting project at https://lnkd.in/eVq-2Faa #hyperinsulinism #raredisease #congenitalhyperinsulinism #hypoglycemia | Hyperinsulinism | Congenital | HI | Hypoglycemia | Rare Disease
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🔊New podcast klaxon🔊 The steatotic liver disease nomenclature was published last year to much fanfare, but what prognostic information does it convey? How do clinical outcomes differ between #MASLD, #MetALD, and #ALD subclasses? For this episode I spoke to Mads Israelsen, first author on a new paper exploring the nomenclature in a Danish cohort of patients with a history of excessive alcohol intake. We cover👇 - Why the need for a new nomenclature replacing #NAFLD? - Study design and participants - Key results, including the stepwise increase in the risk of hepatic decompensation and overall mortality from MASLD, through MetALD, to ALD - What the findings mean for patients, clinicians, and triallists as we enter a new era of therapeutics Listen here or search on your preferred platform: https://lnkd.in/eWZkRZ5Y Read the full paper: https://lnkd.in/ewc-kJPM And the brilliant linked comment by Fredrik Åberg: https://lnkd.in/egKEcQkp Aleksander Krag Nikolaj Torp Camilla Dalby Hansen Katrine Thorhauge Johanne Kragh Hansen Charlotte Wernberg Peter Andersen Katrine Prier Lindvig Emmanuel Tsochatzis Maja Thiele Maru/Mary Rinella
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#NEW from #CTAD2023 | The future of #Alzheimer’s disease diagnosis: #bloodbiomarkers and #amyloid-PET 🎥 🧠 Robin Wolz, PhD, IXICO, shares his thoughts on the next steps for #amyloid-PET #research and implementation. Amyloid-PET has long been considered the gold standard approach for #Alzheimersdisease diagnosis and while it offers high accuracy, its use is limited by cost and availability 🔦 How best to utilize #blood-based #biomarkers alongside PET to minimize cost and invasiveness is a key area of investigation. The introduction of #blood-based #biomarkers has the potential to revolutionize the diagnostic and prognostic work-up of AD, enabling timely and accurate diagnosis whilst keeping PET requirement to a minimum 🩸🌟 Find out more 👉 https://lnkd.in/eDb2m2pM #Dementia
The future of Alzheimer’s disease diagnosis: blood biomarkers and amyloid-PET | VJDementia
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Advancing Rare Disease Research with MILO! Less than 10% of rare diseases have effective treatments, leaving over a billion patients and caregivers in desperate need. But there's hope on the horizon! • Revolutionizing rare disease research • Empowering patients and researchers • Real-world impact through data integration Our team at Milo-Healthcare.com is proud to introduce MILO DCT, a groundbreaking solution revolutionizing rare disease research. Our decentralized clinical trial tools are empowering researchers and patients alike, making trials more feasible and efficient than ever before. From simplifying recruitment with MILO eConsent to enhancing participation with MILO ePRO and EDC, we're reshaping the future of clinical trials. And with real-world data integration, we're turning insights into action, driving progress forward in the fight against rare diseases. Discover the full details of how MILO is transforming rare disease research in our article available here: https://lnkd.in/d7zGj8BU Join us in our mission to transform the landscape of rare disease research. Explore the full details of how MILO DCT is making a difference on Milo-healthcare.com. Together, we can bring hope and healing to those who need it most. #RareDisease #ClinicalTrials #MILOHealthcare
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[ERA Congress - European Renal Association] Our team, Marine Dancer, Bade Kaya, Carolina Oliva Garcia, PhD, MBA, will be present at the 61st ERA Congress, which runs until 26 May in Stockholm (Sweden). We will be delighted to welcome you on our booth B.425 and answer any questions you may have about the contribution of the exome in unknown kidney disease. #Nephrogenomics #Exome #MUC1 #EurofinsTransplantDiagnostics
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If you enjoyed last week's The Genetics Podcast on the potential of engineered tRNA to address rare and ultra-rare diseases, you might be interested in this report, which sheds lights on the complexities of rare disease patient recruitment. 🌟 From tackling participant scarcity to breaking down engagement barriers, we offer strategic solutions to transform your approach to clinical trials.👇 https://lnkd.in/e7vv5QKP #ClinicalTrials #PrecisionMedicine #RareDisease
Navigating rare disease recruitment
sanogenetics.com
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Yesterday was a virtual kickoff of EDC-MASLD EU project, which is coordinated by Örebro University (Tuulia Hyötyläinen and myself), which aims to elucidate the impact of #EndocrineDisruptors on the initiation and progression of metabolic dysfunction-associated steatotic liver disease #MASLD. Looking forward to F2F mtg in Örebro in April, and to work with a great interdisciplinary team! #exposome #metabolomics #AdverseOutcomePathway ... and much more
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To mark Rare Disease Day 2024, this week we are exploring four critical elements to sustaining rare disease innovation in Europe. Today focuses on requirement #1: Great science. The fact that 95% of approximately 6,000-10,000 known rare diseases still lack any approved treatment is down to a combination of clinical and economic hurdles to innovation. First and foremost, innovation requires excellent science – without which there is no question of industry investment in developing and launching a novel therapy. Scientific discovery is progressive and often difficult to predict. Basic research – often supported by public funding and performed in academic institutions – plays an essential role in generating the foundational scientific understanding and technological capability which form the building blocks for future drug development. Industry generally enters the stage once scientific leads are established to transform original science into a clinically tested and approved product, in what is invariably a long and risky process. In Isabelle Laurence and colleagues' recent publication exploring the factors which drive drug development, we examine how rarity exacerbates the scientific challenges to innovation. Indeed, with what little is known about most rare diseases and their populations, great underlying science is even more important, and challenging, to attain. Public policy plays a critical role in driving scientific discovery to expand the field of clinically viable areas for innovation. Though, in isolation, this is not enough to drive rare disease development in Europe, it is an essential pre-requisite. Read on to learn more: https://bit.ly/42UC0Uf #RareDiseaseDay2024 #RareDiseaseDay #RDD2024 #LightUpForRare #ShareYourColours #RareDiseases #RareDiseaseAwareness #RareDiseaseInnovation #Dolon
What does it take to see continued rare disease innovation in Europe?
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Startup Founder | Empowering Science, One Tube at a Time | BiomLife® Ensures Microbial Vitality for Breakthrough Discoveries!
🔬 Exciting Advances in Disease Diagnosis with Biomlife by Ruhvenile® (www.ruhvenile.com)! 🔬 I'm thrilled to share the latest breakthrough in disease diagnosis with Biomlife! 🌐💡 Biomlife is at the forefront of revolutionizing healthcare by leveraging cutting-edge technologies to enhance disease detection and diagnosis. 🚀 Whether it's early cancer detection, infectious diseases, or chronic conditions, Biomlife's innovative solutions are making a significant impact. Key Features: ✅ Precision Medicine: Tailoring treatments based on individual patient characteristics. ✅ Data: Generate your own Biobank with BiomLife and analyse the specimen whenever you like with 2 years. ✅ Early Detection: Identifying diseases at their earliest stages for better prognosis. Our commitment to advancing healthcare is unwavering, and Biomlife is poised to reshape the landscape of disease diagnosis. Join us in the mission to make a positive impact on global health. Curious to learn more? Visit our website www.ruhvenile.com, email to info@ruhvenile.com or reach out for a conversation +91 8587033367. Let's revolutionize healthcare together! 💪🏥 #Biomlife #DiseaseDiagnosis #HealthcareInnovation #Biotechnology #PrecisionMedicine #microbiomeresearch #hospital
